New Gene Therapy Method to
help Muscular Dystrophy – Muscle cells rebuilt in Mice Study
December 26th, 2005
University Medical Center
In a press release from Stanford
University Medical Center, a new gene therapy technique that was
originally intended for skin disease and hemophilia may also have
promise for treating muscular dystrophy. The study conducted at
Stanford University School of Medicine, investigated the dystrophin gene
in hopes to develop a new treatment for people with muscular dystrophy.
The gene therapy technique is called
Rando, named after Thomas Rando MD, PhD an associated professor of
neurology and neurological sciences who has been in the process of try
to find a cure with gene therapy for muscular dystrophy. The obstacle
has been getting the genes to go into all the muscle cells in the body.
The second challenge is to get the cells to permanently manufacture the
beneficial protein created from the gene therapy.
Carmen Bertoni, PhD and Michele
Calos, PhD and associate professor of genetics developed the Rando gene
therapy technique. The technique offers the potential for a long-term
treatment for many genetic diseases, including muscular dystrophy.
Muscular dystrophy is a life
shortening and debilitating disease. The muscle cells break down and
are slowly replaced by fat. People that have muscular dystrophy are
usually confined to a wheelchair and die in their 20’s. At this time
there is no cure, but the potential for the use of gene therapy might
actually change the course of the disease.
The complete study will be published
in the upcoming online edition of the Proceedings of the National
Academy of Sciences during the week of Jan. 2nd, 2006.
In the paper Bertoni used a standard
gene therapy method to establish dystrophin gene, and another gene that
makes a protein glow in mice with muscular dystrophy. The mice that did
not produce enough dystrophin had glowing protein leak out of the cell
slowly. This was a sign that the cell is not healed. However, when she
used Calos’ gene therapy method to establish the same genes, the muscle
cells maintained high levels of dystrophin along the length of the cell
and the glowing protein did not leak and remained inside the cell. The
thought is that the dystrophin actually repaired the muscle that was
"I think our approach has a lot of
potential to overcome issues that have slowed the field of gene
therapy," Calos said. Both Calos and Rando mention that the gene
therapy cure is still far from being achieved, but Calos is self-assured
that her method looks promising and hope it will lead to the cure of
hemophilia, skin disease, epidermolysis bullosa as well as muscular
What makes her approach different is
that the gene is inserted into the cell’s own DNA, making the correction
permanent. The other benefit to her approach is by not using a virus to
disperse the DNA which could have cancer and immune system side
effects. This approach utilized a naked DNA that is able to travel
through the bloodstream to the cells.
Best Syndication Staff Writer
common misspellings: mucular distrophy musculer dystophy muscler
distophy gene jean gen genetic theripy therape